(19) |
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(11) |
EP 4 249 472 A3 |
(12) |
EUROPEAN PATENT APPLICATION |
(88) |
Date of publication A3: |
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13.12.2023 Bulletin 2023/50 |
(43) |
Date of publication A2: |
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27.09.2023 Bulletin 2023/39 |
(22) |
Date of filing: 27.05.2016 |
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(51) |
International Patent Classification (IPC):
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(84) |
Designated Contracting States: |
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AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL
NO PL PT RO RS SE SI SK SM TR |
(30) |
Priority: |
30.05.2015 US 201562168726 P
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(62) |
Application number of the earlier application in accordance with Art. 76 EPC: |
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16804178.8 / 3310169 |
(71) |
Applicant: PTC Therapeutics, Inc. |
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South Plainfield, NJ 07080 (US) |
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(72) |
Inventors: |
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- NARYSHKIN, Nikolai, A.
East Brunswick, NJ 08816 (US)
- DAKKA, Amal
Whitehouse Station, NJ 08889 (US)
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(74) |
Representative: Jones Day |
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Rechtsanwälte, Attorneys-at-Law, Patentanwälte
Prinzregentenstrasse 11 80538 München 80538 München (DE) |
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(54) |
METHODS FOR MODULATING RNA SPLICING |
(57) In one aspect, described herein is a recognition element for splicing (REMS) that
can be recognized by a compound provided herein. In another aspect, described herein
are methods for modulating the amount of a product of a gene, wherein a precursor
RNA transcript transcribed from the gene contains a REMS, and the methods utilizing
a compound described herein. More particularly, described herein are methods for modulating
the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor
RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing
a compound described herein. In another aspect, provided herein are artificial gene
construct comprising a REMS, and uses of those artificial gene constructs to modulate
functional protein production. In another aspect, provided herein are methods for
altering endogenous genes to comprise a REMS, and the use of a compound described
herein to modulate the functional protein produced from such altered endogenous genes.